Almost 1/3 of the Warning Letters on FDA’s website pertaining to medical foods have been issued within the last year.1 The most recent examples illustrate that FDA is enforcing the principles set forth in its draft Guidance on Frequently Asked Questions about Medical Foods, Second Edition2.

The extended comment period on the draft Guidance ended December 16, 2013 and ten days later, on December 26, 20013, FDA issued Warning Letters to NVN Therapeutics and Accera Inc., that incorporate the concepts of the draft guidance. Notably, the Warning Letters further narrow the definition of medical foods set forth in the regulations3 to mean foods that are specially formulated and processed (as opposed to a naturally occurring foodstuff used in a natural state) “for a patient who is seriously ill or who requires use of the product as a major component of a disease or condition’s specific dietary management.” This language is directly from page 4 of the draft medical food guidance and appears nowhere in the regulations or in the statutory definition of a medical food.4

When one takes into account FDA’s other major draft guidance that affects medical foods, it appears that the drug definition and drug requirements may well overtake the Congressional language placing medical foods outside the drug definition. The FDA’s draft Guidance for Clinical Investigators, Sponsors, and IRBs, Investigational New Drug Applications (INDs) — Determining Whether Human Research Studies Can Be Conducted Without an IND (September 2013)5 describes the conditions under which research into the benefits of medical foods on patient populations would require an Investigational New Drug (IND) submission. In that document, the “FDA regulates conventional foods (including infant formula) that are intended to affect the structure or function of the body as foods, not drugs, as long as the intended structure or function effect derives from the product’s character as a food — its taste, aroma, or nutritive value. A clinical investigation intended only to evaluate the nutritional effects of a food (including medical foods) would not require an IND, but if the product is no longer being consumed as a food — primarily for taste, aroma, or nutritive value — but used as a drug for some other physiological effect or the investigation intended to evaluate other effects of a food on the structure or function of the body would require an IND.” The line between medical food and drug research appears less than clear. 

In the meantime the status of medical foods is being defined now in Warning Letters and draft guidances. Without a concerted response by the industry, the future of new and existing medical foods is likely to be substantially altered and patient access to medical foods as a component of medical therapy significantly reduced.